Nemaline myopathy
MONDO:0018958Nemaline myopathy (NM) encompasses a large spectrum of myopathies characterized by hypotonia, weakness and depressed or absent deep tendon reflexes, with pathologic evidence of nemaline bodies (rods) on muscle biopsy.
Also known as: NEM, NM, nemaline body disease, nemaline myopathy, nemaline rod myopathy, rod myopathy, Rod body disease, Rod-body myopathy
17 clinical trials for this condition and its sub-types, 13 tagged with Nemaline myopathy itself.
Follow this condition to get notified about new trialsWhere it sits in the disease tree
Browse by category →Sub-types of Nemaline myopathy
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Nemaline myopathy 5 2 trials
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Childhood-onset nemaline myopathy 1 trial · 2 incl. sub-types
6 sub-types
- Nemaline myopathy 6 1 trial
- Congenital myopathy 23 0 trials
- Congenital myopathy 2a, typical, autosomal dominant 0 trials
- Congenital myopathy 4B, autosomal recessive 0 trials
- Nemaline myopathy 2 0 trials
- Nemaline myopathy 9 0 trials
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Severe congenital nemaline myopathy 0 trials · 1 incl. sub-types
5 sub-types
- Nemaline myopathy 8 1 trial
- Congenital myopathy 2a, typical, autosomal dominant 0 trials
- Nemaline myopathy 10 0 trials
- Nemaline myopathy 2 0 trials
- Nemaline myopathy 9 0 trials
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MYPN-related myopathy 0 trials
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Adult-onset nemaline myopathy 0 trials
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Typical nemaline myopathy 0 trials
6 sub-types
- Congenital myopathy 23 0 trials
- Congenital myopathy 2a, typical, autosomal dominant 0 trials
- Nemaline myopathy 10 0 trials
- Nemaline myopathy 2 0 trials
- Nemaline myopathy 7 0 trials
- Nemaline myopathy 9 0 trials
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Eye implant could replace monthly shots for blinding disease
Disease control OngoingThis study tests a small, refillable implant placed in the eye that slowly releases medication for wet age-related macular degeneration (nAMD). It aims to see if refilling the implant every 24 weeks works as well as getting a standard eye injection every 4 weeks. About 68 Chinese…
Phase 3 • Sponsor: Hoffmann-La Roche • Aim: Disease control
Last updated Sep 03, 2026 00:00 UTC
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One-Shot gene therapy could end monthly eye injections for wet AMD
Disease control OngoingThis study tests a one-time gene therapy called RGX-314 for wet age-related macular degeneration (AMD), a leading cause of vision loss. About 714 adults aged 50-89 who currently need regular eye injections will receive either the gene therapy or standard treatment. The goal is to…
Phase 3 • Sponsor: AbbVie • Aim: Disease control
Last updated Aug 05, 2026 00:00 UTC
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Gene therapy could end frequent eye injections for wet AMD
Disease control OngoingThis study tests a one-time gene therapy called RGX-314 for wet age-related macular degeneration (AMD), a leading cause of vision loss. The treatment aims to help the eye produce its own medicine to stop leaky blood vessels, potentially replacing the need for repeated eye injecti…
Phase 2/3 • Sponsor: AbbVie • Aim: Disease control
Last updated Jun 27, 2026 12:24 UTC
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UK study tracks rare muscle disease to pave way for future treatments
Knowledge-focused OngoingThis study follows 45 people with nemaline myopathy in the UK to learn how the disease naturally progresses. Researchers collect medical exams, quality-of-life surveys, and physical assessments over time. The goal is to identify the best ways to measure the disease in future clin…
Sponsor: University of Oxford • Aim: Knowledge-focused
Last updated Jun 27, 2026 12:07 UTC